Gene therapy
AssigneesOspedale San Raffaele S.R.L., Fondazione Telethon
InventorsPietro Genovese, Giulia Schiroli, Luigi Naldini, Aurelien Jacob, Samuele Ferrari
Life of this patent
Estimating the term…
Abstract
An agent which promotes homology directed DNA repair for use in haematopoietic stem and/or progenitor cell gene therapy, wherein said haematopoietic stem cells are gene edited. Use of an agent which promotes homology directed DNA repair, for increasing the survival and/or engraftment of gene edited haematopoietic stemand/or progenitorcells or for increasing the efficiency of gene editing of haematopoietic stem and/or progenitor cells.
Claims64
An agent which promotes homology directed DNA repair for use in haematopoietic cell gene therapy, haematopoietic stem cell gene therapy, and/or haematopoietic progenitor cell gene therapy, wherein said gene therapy comprises gene editing.
An agent which promotes homology directed DNA repair, for use in increasing the survival and/or engraftment of gene edited haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells.
Use of an agent which promotes homology directed DNA repair, for increasing the survival and/or engraftment of gene edited haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells, or for increasing the efficiency of gene editing of haematopoietic cells, haematopoietic stem cells, and/or haematopoietic progenitor cells.
A method of gene editing a population of haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells comprising the steps:
- (a) contacting the population of haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells with an agent which promotes homology directed DNA repair;
- (b) introducing gene editing machinery to the population of haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells with one or more vectors;
- and
- (c) editing the genome of said haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells.
The method of claim 22, wherein the inhibitor of p53 activation is a p53 dominant negative peptide, an ataxia telangiectasia mutated (ATM) kinase inhibitor or an ataxia telangiectasia and Rad3-related protein (ATR) inhibitor.
An AAV vector comprising a barcode sequence.
Use of a barcode sequence for clonal tracking of cells.
Dependencies are inferred from the claim text.